Abstract
Disclosed herein are systems for modifying nucleic acid splicing of a target RNA such as an SCN1A mRNA. The system may be useful in a method such as a method for treating a genetic disorder such as epilepsy. The system may include an expression system, or a modified small nuclear RNA (snRNA) that includes an exonic splicing silencer (ESS) sequence, and an antisense nucleic acid sequence that targets the target RNA.
Full Text
What is claimed is:
Disclosed herein are systems for modifying nucleic acid splicing of a target RNA such as an SCN1A mRNA. The system may be useful in a method such as a method for treating a genetic disorder such as epilepsy. The system may include an expression system, or a modified small nuclear RNA (snRNA) that includes an exonic splicing silencer (ESS) sequence, and an antisense nucleic acid sequence that targets the target RNA.
Timeline
Filed
04/01/2026Published
07/23/2026Granted
Not AvailableIPC Codes(5)
C12N 15/113:Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 38/17:from animals; from humans
A61K 48/00:Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy